Efficacy and Safety of Bimagrumab/BYM338 at 52 Weeks on Physical Function, Muscle Strength, Mobility in sIBM Patients

Sporadic Inclusion Body Myositis Clinical Trial

— RESILIENT  

Official title:

A Randomized, Double-blind, Placebo-controlled, Multicenter, Parallel Group, Dose-finding, Pivotal, Phase 2b/3 Study to Evaluate the Efficacy, Safety, and Tolerability of Intravenous BYM338 at 52 Weeks on Physical Function, Muscle Strength, and Mobility and Additional Long Term Safety up to 2 Years in Patients With Sporadic Inclusion Body Myositis

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01925209
• Other study ID #: CBYM338B2203
• Status: Completed
• Phase: Phase 2/Phase 3
• First received: August 15, 2013
• Last updated: August 7, 2017
• Start date: September 26, 2013
• Est. completion date: January 6, 2016

Study information

• Verified date: August 2017
• Source: Novartis
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

This study evaluated the efficacy, safety and tolerability of multiple doses of bimagrumab/BYM338 vs placebo, when administered intravenously (i.v.), on physical function, muscle strength, and mobility in patients with sporadic inclusion body myositis (sIBM).

Recruitment information / eligibility

• Status: Completed
• Enrollment: 251
• Est. completion date: January 6, 2016
• Est. primary completion date: January 6, 2016
• Accepts healthy volunteers: No
• Gender: All
• Age group: 36 Years to 85 Years

Eligibility

Key Inclusion Criteria:

• Diagnosed with sporadic inclusion body myositis;

• Must be able to walk (assistive aids allowed, including intermittent use of wheelchair);

Key Exclusion Criteria:

• Must not have other conditions that significantly limit ability to move around;

• Must not be using corticosteroids. Must not have used systemic corticosteroid (at daily dose >=10mg prednisone) for the past 3 months;

• Must meet cardiovascular requirements;

• Must not be pregnant or nursing;

• Must not have a chronic active infection (e.g., HIV, hepatitis B or C, tuberculosis, etc.);

Related Conditions & MeSH terms

• Myositis
• Myositis, Inclusion Body
• Sporadic Inclusion Body Myositis

Intervention

Drug:
• BYM338/bimagrumab
BYM338, a 150 mg/mL concentrate for solution for i.v. infusion, was provided in colorless glass vials with a rubber stopper and aluminum flip-off caps.
• Placebo
Matching placebo to BYM338 was provided in colorless glass vials with a rubber stopper and aluminum flip-off caps.

Locations

Country	Name	City	State
Australia	Novartis Investigative Site	Cauldfield	Victoria
Australia	Novartis Investigative Site	Nedlands	Western Australia
Australia	Novartis Investigative Site	St. Leonards	New South Wales
Belgium	Novartis Investigative Site	Bruxelles
Belgium	Novartis Investigative Site	Edegem
Belgium	Novartis Investigative Site	Gent
Denmark	Novartis Investigative Site	Copenhagen
France	Novartis Investigative Site	Paris
Italy	Novartis Investigative Site	Brescia	BS
Italy	Novartis Investigative Site	Messina	ME
Italy	Novartis Investigative Site	Milano	MI
Italy	Novartis Investigative Site	Padova	PD
Italy	Novartis Investigative Site	Roma	Lazio
Japan	Novartis Investigative Site	Kodaira-city	Tokyo
Japan	Novartis Investigative Site	Kumamoto City	Kumamoto
Japan	Novartis Investigative Site	Nagoya-city	Aichi
Japan	Novartis Investigative Site	Osaka-city	Osaka
Japan	Novartis Investigative Site	Sendai-city	Miyagi
Japan	Novartis Investigative Site	Tokushima-city	Tokushima
Japan	Novartis Investigative Site	Wakayama-city	Wakayama
Netherlands	Novartis Investigative Site	Amsterdam
Netherlands	Novartis Investigative Site	Leiden
Switzerland	Novartis Investigative Site	Zuerich
United Kingdom	Novartis Investigative Site	London
United Kingdom	Novartis Investigative Site	Newcastle upon Tyne
United Kingdom	Novartis Investigative Site	Salford	Manchester
United States	Novartis Investigative Site	Baltimore	Maryland
United States	Novartis Investigative Site	Boston	Massachusetts
United States	Novartis Investigative Site	Boston	Massachusetts
United States	Novartis Investigative Site	Columbus	Ohio
United States	Novartis Investigative Site	Dallas	Texas
United States	Novartis Investigative Site	Houston	Texas
United States	Novartis Investigative Site	Kansas City	Kansas
United States	Novartis Investigative Site	Miami	Florida
United States	Novartis Investigative Site	Orange	California
United States	Novartis Investigative Site	Phoenix	Arizona
United States	Novartis Investigative Site	Portland	Oregon
United States	Novartis Investigative Site	Sacramento	California

Sponsors (1)

Lead Sponsor	Collaborator
Novartis Pharmaceuticals

Countries where clinical trial is conducted

United States,  Australia,  Belgium,  Denmark,  France,  Italy,  Japan,  Netherlands,  Switzerland,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change From Baseline in 6 Minute Walking Distance (6MWD) Test at Week 52	The 6MWD test measured the distance (in meters) that a participant walked in a 6 minute timeframe. A positive change from baseline indicates improvement.	Baseline, Week 52
Secondary	Estimated Within Treatment Group Lean Body Mass (LBM) Ratio at Week 52	LBM was measured via dual energy x-ray absorptiometry (DXA) and calculated as (LBM at Week 52/LBM at baseline)*100 . A positive change from baseline indicates improvement.	Baseline, Week 52
Secondary	Change From Baseline in Quadriceps Quantitative Muscle Testing (QMT) on the Right Side at Week 52	Quadriceps muscle strength was measured by portable fixed dynamometry (PFD) on the right side. A negative change from baseline indicates deterioration.	Baseline, Week 52
Secondary	Change From Baseline in Sporadic Inclusion Body Myositis (sIBM) Functional Assessment (sIFA) Score at Week 52	Self-reported physical function was assessed by a newly developed patient reported outcome named sporadic inclusion body myositis (sIBM) functional assessment (sIFA). The sIFA consists of 11 items scored on an 11 point numerical rating scale from 0 (no difficulty) to 10 (unable to do) across 3 domains: upper body functioning, lower body functioning and general functioning. Participants completed the assessment where the recall period was the past week prior to completing the patient reported outcome (PRO). The total score on the sIFA scale ranges from 0 (minimum) to 110 (maximum). Higher values represent a worse outcome. A positive change from baseline indicates deterioration.	Baseline, Week 52
Secondary	Estimated Annual Number of Falls Per Patient Within Treatment Group	Participants documented any fall occurrences in a paper diary during the study.	Week 52
Secondary	Change From Baseline in Short Physical Performance Battery (SPPB) Score at Week 52	The SPPB evaluated lower extremities function by testing gait speed, ability to keep standing balance and time to rise from a chair five times. The sub-score for each test ranged from 0 to 4. The summary score, which was a summation of scores from the 3 tests, ranged from 0 to 12. An increase in score indicates improvement in physical performance. A negative change from baseline indicates deterioration.	Baseline, Week 52

External Links

•   Results for CBYM338B2203 from the Novartis Clinical Trial Results Website